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Nephrotic SyndromePhase 2NCT02592798

Pilot Study to Evaluate the Safety and Efficacy of Abatacept in Adults and Children 6 Years and Older With Excessive Loss of Protein in the Urine Due to Either Focal Segmental Glomerulosclerosis (FSGS) or Minimal Change Disease (MCD)

A Phase 2 study testing Abatacept, Normal Saline and D5W in people with Nephrotic Syndrome and Focal Segmental Glomerulosclerosis, run by Bristol-Myers Squibb at 27 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

The purpose of this study is evaluate if abatacept is effective and safe in decreasing the level of protein loss in the urine in patients with excessive loss of protein in the urine (nephrotic syndrome) due to either focal segmental glomerulosclerosis (FSGS) or minimal change disease (MCD). Read more about this study

Who is it for?
Ages 6 years and older
What is being tested?
Abatacept, Normal Saline and D5W
Type of treatment
Injection or infusion
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
36 participants
Where
27 sitesUnited States
Study dates
9 Mar 2016to 28 Jan 2020
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 6 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Male and female subjects ages ≥ 6 years
  • Subjects resistant to corticosteroids, calcineurin inhibitors (cyclosporine and tacrolimus), sirolimus, mycophenolate mofetil (MMF), mycophenolic acid (MPA), or cyclophosphamide or intolerant to at least 2 of these
  • UPCR ≥ 3 at screening
  • FSGS or MCD confirmed by renal biopsy
  • eGFR ≥ 45 for children and adults
  • Concomitant use of angiotensin-converting-enzyme inhibitor (ACEi) or angiotensin receptor blocker (ARB) at stable doses for at least 2 weeks or have intolerance documented in the source documents maintained at the site

Who cannot join

  • Kidney diseases other than FSGS or MCD
  • Collapsing FSGS
  • Systemic lupus erythematosus
  • Diabetes mellitus, both type 1 and type 2
  • Clinically significant congestive heart failure
  • Post renal transplantation, including relapsing post-transplant FSGS
  • Body mass index (BMI): > 40 in subjects ≥ 18 years of age and ≥ 99% percentile for subjects < 18 years of age
  • Other protocol defined inclusion/exclusion criteria may apply

Trial locations and contacts

27 sites in 1 country.

27 sites in 1 country

United States27 sites

  • University of Michigan

    Ann Arbor, Michigan, 48109

  • Emory University

    Atlanta, Georgia, 30322-1015

  • University Of Colorado Denver

    Aurora, Colorado, 80045

  • NIH Clinical Center - NIDDK

    Bethesda, Maryland, 20892

  • The University Of Alabama At Birmingham

    Birmingham, Alabama, 35233

  • University of Alabama-Birmingham-Parent Account

    Birmingham, Alabama, 35294

  • Boston Childrens Hospital

    Boston, Massachusetts, 02115

  • Brigham And Women'S Hosp Inc.

    Boston, Massachusetts, 02115

  • Levine Children's Hospital

    Charlotte, North Carolina, 28203

  • Cincinnati Children'S Hospital Medical Center

    Cincinnati, Ohio, 45229-3039

  • The Metro Health System

    Cleveland, Ohio, 44109

  • Cleveland Clinic

    Cleveland, Ohio, 44195

  • The Ohio State University Wexner Medical Center

    Columbus, Ohio, 43210

  • Renal Disease Research Institute

    Dallas, Texas, 75235

  • University Of Texas Southwestern Medical Center

    Dallas, Texas, 75390

  • Duke University

    Durham, North Carolina, 27701

  • Childrens Mercy Hospital

    Kansas City, Missouri, 64108

  • Loyola University Medical Center

    Maywood, Illinois, 60153

  • University Of Miami Miller School Of Medicine

    Miami, Florida, 33136

  • New York University Langone Medical Center

    New York, New York, 10016

  • Columbia University Medical Center (Cumc)

    New York, New York, 10032

  • Nemours Childrens Hospital

    Orlando, Florida, 32827

  • University Of Pennsylvania

    Philadelphia, Pennsylvania, 19104

  • Local Institution

    Pittsburgh, Pennsylvania, 15224

  • Mayo Clinic

    Rochester, Minnesota, 55905

  • Los Angeles Biomedical Research Institute

    Torrance, California, 90502

  • Children's National Health System

    Washington D.C., District of Columbia, 20010

Other trials in United States

About this study

Official title: A Phase II Randomized, Placebo-Controlled, Double-Blind, Parallel Arms, Pilot Study to Evaluate the Efficacy and Safety of Intravenous Abatacept in Treatment Resistant Nephrotic Syndrome (Focal Segmental Glomerulosclerosis/ Minimal Change Disease)

The purpose of this study is evaluate if abatacept is effective and safe in decreasing the level of protein loss in the urine in patients with excessive loss of protein in the urine (nephrotic syndrome) due to either focal segmental glomerulosclerosis (FSGS) or minimal change disease (MCD). Candidates must have a prior kidney biopsy with either diagnosis. Another kidney biopsy will not be required as part of the study. Candidates must have failed or be intolerant of prior therapy for their kidney disease. The failed or intolerant therapy must include corticosteroids and at least one other drug. Candidates can be adults and children over the age of 6. Abatacept will be administered by venous infusion every 4 weeks.

Being tested
Abatacept, Normal Saline, D5W
Medical subject headings
Nephrotic Syndrome, Glomerulosclerosis, Focal Segmental, Nephrosis, Lipoid

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • AbataceptDrug

    Abatacept IV administered on Day 1, 15, 29 and then every 28 days

  • Normal SalineOther

    Normal Saline administer on Day 1, 15, 29 and then every 28 days

  • D5WOther

    Also called 5% Dextrose in Water

    Dextrose 5% in Water (D5W) administered on Day 1, 15, 29 and then every 28 days

Study groups in NCT02592798
Study groupRoleWhat they receive
AbataceptExperimental* Double Blind Periods 1 and 2 (DB1 and DB2): Abatacept IV administered on Day 1, 15, 29 and then every 28 days until the end of the Double Blind Period. * Open Label Period (OLE): Abatacept IV administered every 28 days
Receives: Drug: Abatacept
PlaceboPlacebo comparator* Double Blind Periods 1 and 2 (DB1 and DB2): Normal Saline or Dextrose 5% in Water (D5W) administer on Day 1, 15, 29 and then every 28 days until the end of the Double Blind Period. * Open Label Period (OLE): Abatacept IV administered every 28 days
Receives: Other: Normal Saline; Other: D5W

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Percentage of Participants in Renal Response at Day 113

    Renal Response is defined as the presence of all the following criteria:

    PROTEINURIA: Reduction of baseline urine protein/creatinine ratio (UPCR) of >= 50% and to less than 3.

    RENAL FUNCTION: No worsening of baseline estimated glomerular filtration rate (eGFR) defined as within normal range if normal at baseline or ≥ 75% baseline value if below normal at baseline.

    Measured: From first dose to 113 days following first dose of the indicated period (113 days for Double-Blind Period, 226 days for Open Label Period)

Secondary outcomes (13)

  1. Mean Change From Baseline in Urine Protein/Creatinine Ratio (UPCR) at Day 113

    Measured: From baseline (measured at day 1 of study) to 113 days following first dose administered in the indicated treatment period (113 days for Double-Blind Period, 226 days for Open Label Period)

  2. Mean Change From Baseline in Serum Albumine at Day 113

    Measured: From baseline (measured at day 1 of the study) to 113 days following first dose administered in the indicated treatment period (113 days for Double-Blind Period, 226 days for Open Label Period)

  3. Percentage of Participants Achieving Complete Remission at Day 113

    Complete Remission is defined as the presence of all the following criteria:

    PROTEINURIA: Urine protein/creatinine ratio (UPCR) ≤ 0.3. RENAL FUNCTION: No worsening of baseline estimated glomerular filtration rate (eGFR) defined as within normal range if normal at baseline or ≥ 75% baseline value if below normal at baseline.

    Measured: From first dose to 113 days following first dose of the indicated period (113 days for Double-Blind Period, 226 days for Open Label Period)

  4. Mean Change From Baseline in Patient Reported Outcomes Measurement Information System (PROMIS) at Day 113 - Adult Participants

    PROMIS was used to capture patient reported outcomes relevant to participants with nephrotic syndrome. Mean change from baseline is reported for the following measurements:

    Fatigue: Short From (SF) Fatigue v1.0 Adult 8a, composed of 8 questions, each one scored from 1 (Not at all) to 5 (Very much). Output presented as Total score, ranging from 8 (most desirable outcome) to 40 (least desirable outcome).

    Pain Interference: SF Pain Interference v1.0 Adult 8a, (measuring how much pain is interfering with daily activities). Composed of 8 questions, each one scored from 1 (Not at all) to 5 (Very much). Output presented as Total score, ranging from 8 (most desirable outcome) to 40 (least desirable outcome).

    Physical Function: SF Physical Function v1.2 Adult 8b, composed of 8 questions, each one scored from 1 (Without any difficulty) to 5 (Unable to do). Output presented as Total score, ranging from 8 (most desirable outcome) to 40 (least desirable outcome).

    Measured: From baseline (measured at day 1 of study) to 113 days following first dose administered in the Double-Blind Period

  5. Mean Change From Baseline in Patient Reported Outcomes Measurement Information System (PROMIS) at Day 113 - Pediatric Participants

    PROMIS was used to capture patient reported outcomes relevant to participants with nephrotic syndrome. Mean change from baseline is reported for the following measurements:

    Fatigue: Short From (SF) Fatigue v1.0 Peds 10a, composed of 10 questions, each one scored from 0 (Never) to 4 (Almost Always). Output presented as Total score, ranging from 0 (most desirable outcome) to 40 (least desirable outcome).

    Pain Interference: SF Pain Interference v1.0 Peds 8a, ( measuring how much pain is interfering with daily activities). Composed of 8 questions, each one scored from 0 (Never) to 4 (Almost always). Output presented as Total score, ranging from 0 (most desirable outcome) to 32 (least desirable outcome).

    Mobility: SF Physical Function-Mobility v1.0 Peds 8a, composed of 8 questions, each one scored from 0 (Not able to do) to 4 (With no trouble). Output presented as Total score, ranging from 0 (least desirable outcome) to 32 (most desirable outcome).

    Measured: From baseline (measured at day 1 of study) to 113 days following first dose administered in the Double-Blind Period

  6. Number of Participants Experiencing Adverse Events

    This outcome describes the number of participants experiencing various types of any grade Adverse Events (AEs).

    The Cumulative Abatacept Safety Period starts at the time of the first dose of Abatacept (either in the Double-Blind Period or in the Open Label Period) and lasts 56 days after the last dose of Abatacept

    Measured: From first dose in the indicated period to 56 days following last dose in the indicated period (169 days for the Double-Blind Period, up to 337 days for the Cumulative Abatacept Safety Period)

  7. Number of Participants Experiencing Adverse Events of Special Interest

    Number of participants experiencing various types of Adverse Events of interest, including infections, malignancies, autoimmune disorders, and infusional related reactions.

    Measured: From first dose on day 1 to 56 days following last dose (approximately 330 days)

  8. Percentage of Participants With Positive Antibody Response Relative to Baseline

    A positive response relative to baseline is defined as a positive response at a post-baseline visit that has a titer value greater than the positive baseline titer value. If the baseline titer is missing or negative, any post-baseline positive titer value is considered a positive response relative to baseline.

    Immunogenicity response is presented as the total of immunogenicity response for "CTLA4 and possibly Ig" and "Ig and/or Junction Region".

    Measured: From baseline (day 1) to 168 days following last dose (up to 15 months). Results at day 113 from first dose, day 56, day 84 and day 168 after last dose are presented

  9. Minimum Blood Plasma Concentration (Cmin) of Abatacept - Adult Participants

    Measured: From first dose to 113 days after first dose in the Double Blind Period. Data collected on day 15, day 29, day 57, day 85 and day 113

  10. Minimum Blood Plasma Concentration (Cmin) of Abatacept - Pediatric Participants

    Measured: From first dose to 113 days after first dose in the Double Blind Period. Data collected on day 15, day 29, day 57, day 85 and day 113

  11. Maximum Observed Serum Concentration (Cmax) of Abatacept

    Measured: Day 85 after first dose in the Double Blind Period

  12. Area Under the Serum Concentration Time Curve Over a Dosing Interval (AUC(TAU)) of Abatacept

    Measured: From Day 85 to Day 113 in the Double Blind Period

  13. Time to Reach Peak Serum Concentration (Tmax(h)) of Abatacept

    Measured: Day 85 after first dose in the Double Blind Period

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted30 Oct 2015 (estimated)
  2. Study start9 Mar 2016
  3. Primary completion28 Jan 2020
  4. Study completion28 Jan 2020

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
36 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02592798
Sponsor's protocol ID
IM101-566
Other IDs
2015-005450-36, 2015
Status last verified
Feb 2021
First submitted
29 Oct 2015
Last updated
5 Mar 2021
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Publications

  • Liu ID, Willis NS, Craig JC, Hodson EM. Interventions for idiopathic steroid-resistant nephrotic syndrome in children. Cochrane Database Syst Rev. 2025 May 8;5(5):CD003594. doi: 10.1002/14651858.CD003594.pub7.

    PubMed 40337980

  • Azukaitis K, Palmer SC, Strippoli GF, Hodson EM. Interventions for minimal change disease in adults with nephrotic syndrome. Cochrane Database Syst Rev. 2022 Mar 1;3(3):CD001537. doi: 10.1002/14651858.CD001537.pub5.

    PubMed 35230699

  • Hodson EM, Sinha A, Cooper TE. Interventions for focal segmental glomerulosclerosis in adults. Cochrane Database Syst Rev. 2022 Feb 28;2(2):CD003233. doi: 10.1002/14651858.CD003233.pub3.

    PubMed 35224732

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Abatacept, Normal Saline and D5W in people with Nephrotic Syndrome and Focal Segmental Glomerulosclerosis, run by Bristol-Myers Squibb at 27 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 6 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 5 Mar 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 27 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 5 Mar 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Percentage of Participants in Renal Response at Day 113 (time frame: From first dose to 113 days following first dose of the indicated period (113 days for Double-Blind Period, 226 days for Open Label Period)). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02592798, which also lists 3 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Mar 2021.

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